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Author
Topic: gene therapy good results see this animation how it works (Read 5352 times)

The results were astounding. patient's T-cell levels not only stopped dropping but also dramatically increased in some patients VIRxSYS is currently conducting clinical trials for VRX496. see this animation... we may need to do activism to get this widely available if it works in the study

scientists in a Manhattan laboratory are in the midst of studies that would put the pathogen to noble use.

Gene Therapy Shows Promise Against HIVGene-base therapy may be effective against HIV, U.S. researchers report.An investigational gene-based immunotherapy called VRX496 suggests it can fight the virus, according to a phase I, open-label, non-randomized clinical trial conducted at the University of Pennsylvania School of Medicine.The trial evaluated the safety and tolerability of VRX496 -- a CD4 T cell treatment -- in five people with chronic HIV infection who had all failed to respond to at least two antiretroviral drug regimens.In response to the treatment, the five patients experienced decreases in viral load and showed stable CD4 T cells."This is an important milestone in the development of what we believe will be the next-generation of HIV therapy," reports Riku Rautsola, Ph.D., president and CEO of VIRxSYS Corporation, which is testing the treatment with researchers from the University of Pennsylvania School of Medicine in Philadelphia. "VRX496 is engineered in a way that debilitates HIV's ability to replicate and mutate around the therapy, overcoming a common problem with current drug treatments."VIRxSYS corporation is a private biotech company whose mission is to develop gene therapies using its proprietary lentiviral vector delivery system. The company is in Phase II clinical trials testing the first application of its gene therapy technology against HIV, VRX496. VIRxSYS also has collaborations for therapies in certain cancers and genetic diseases. The ability to safely and efficiently deliver genetic therapies has long been an obstacle to successful gene therapy, but VIRxSYS's lentiviral vector is designed to deliver on the promise of genetic medicine.The Company expects that its genetic medicines will provide dramatically improved patient outcomes, including disease remission and may, possibly, provide permanent cures. While VIRxSYS's initial focus is on cellular therapies for HIV/AIDS, cancer, and genetic diseases, the Company has established collaborations to develop vaccines for those and other diseases. Future applications for the technology could also include countering bioterrorism and advancing the field of functional genomics.

HIV, a member of the group of microbes known as lentiviruses, is an ideal vehicle, scientists say, to ferry corrective genes into cells of patients affected by thalassemia and sickle cell disease. The two related genetic disorders are characterized by red blood cells that are incapable of effectively carrying oxygen.

Research under way at Memorial Sloan-Kettering Cancer Center is aimed at incorporating corrective DNA into patients' chromosomes, allowing their red blood cells to effectively carry oxygen to tissues.

Medical experts hope to use HIV as the vector -- or the vehicle -- that carries DNA into patients' cells. The virus is first stripped of all genes that cause deadly infection. Those, however, that allow it to insinuate itself neatly into a person's chromosomes are left in place.

Dr. Michel Sadelain, director of gene transfer and somatic cell engineering at the cancer center, said in the early 1990s that even he was leery about the therapeutic potential of a lethal virus. But molecular biologists continued to demonstrate, Sadelain said, that Mother Nature had developed viruses as the most efficient entities capable of integrating themselves into the chromosomes.

"Multiple scientists have been working on this virus for more than two decades," he said yesterday. "Compared with other members of the lentivirus family, we know 100 times more about HIV because it causes a disease. And even though this may seem to be a paradox to some, we also know that it is so remarkably efficient in the way it introduces itself into cells.

"That is when it suddenly dawns on you that it should be valuable in clinical applications," Sadelain said.

The first inklings that HIV could be used as a gene therapy vector, he said, occurred in the early 1990s. "My own instinct at the time was: 'No forget it,'" Sadelain said. "But our first paper came out in 1996, which showed that [an HIV] vector could be engineered."

Earlier this month, a team at the University of Pennsylvania demonstrated that disabling HIV's infection-causing machinery indeed creates an ideal vector to transport genes into patients' cells -- even those infected with HIV itself.

Dr. Bruce Levine, associate professor of pathology and laboratory medicine at the University of Pennsylvania's Abramson Cancer Center, used an HIV vector to carry immune-boosting genes into the cells of patients infected with HIV. Although the experimental treatment may at first seem mind-boggling, the vector, because it is disabled, cannot cause further disease.

Levine's research is the first to prove that HIV can have clinical benefits. The Phase I clinical trial was reported earlier this month in the Proceedings of the National Academy of Sciences. The five patients who underwent this form of gene therapy were resistant to all HIV drugs.

"There was debate about what would be the appropriate disease" for this form of therapy, Levine said yesterday, adding the U.S. Food and Drug Administration recommended "that it be a patient population that has no other treatment options."

With the gene on board the HIV vector, the treatment "throws a monkey wrench in the natural HIV's life cycle," Levine said of a therapy that uses a disabled HIV as part of a treatment regimen to rescue patients who otherwise were doomed to death.

"Someone once said to me that this seems like magic," Levine said. "My response is that science is about making the magic possible."

HIV, the virus that causes AIDS, attacks T-cells which are white blood cells that are critical to the immune system.

In the first in-human trial ever, Bruce Levine and Carl June of the Department of Pathology and Laboratory Medicine, along with Penn Medicine professor Rob Roy MacGregor, took T-cells from HIV patients and inserted a modified virus back into them.

Levine described the engineered virus as "wearing the enemy's uniform."

"The use of HIV itself ... has never been done before," he said.

The trio recently published the results of the first phase of their study, in which they observed the effects of a single injection.

"The modified virus tricks its way into our cells," MacGregor said.

The results were astounding. June said patient's T-cell levels not only stopped dropping but also dramatically increased in some patients.

The primary objective of the study was to determine the "safety and feasibility" of the procedure, Levine said.

There are high risks associated with such treatment, June said, including leukemia and the possibility of the virus mutating and making the patient sicker.

MacGregor said the five patients that participated in the study had advanced HIV that had failed to respond to other therapy.

They were, in a sense, "backed into a corner," MacGregor said, with nowhere else to turn.

"It's an altruistic cohort of people," Levine said.

It took years for June and Levine to get approval for their study, especially after the death of 18-year-old Jesse Gelsinger from complications of a separate gene-therapy study conducted at Penn in 1999.

The field of gene therapy underwent "intense scrutiny" as a result, June said. He added that, while "appropriate," it put them "on hold for about two years."

If the studies continue to prove safe and effective, the question becomes whether this can be a treatment for the larger HIV-infected population.